voretigene neparvovec-rzyl

BIOLOGICAL2 trials

Sponsors

Spark Therapeutics, Inc.

Conditions

Leber Congenital Amaurosis

Phase 1

Safety Study in Subjects With Leber Congenital Amaurosis

CompletedNCT00516477

Spark Therapeutics, Inc.Leber Congenital Amaurosis

Start: 2007-09-30End: 2018-03-20Target: 12Updated: 2020-11-02

Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2

Active, not recruitingNCT01208389

Spark Therapeutics, Inc.Leber Congenital Amaurosis

Start: 2010-11-30End: 2027-10-31Target: 12Updated: 2025-04-29

Related Papers

Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial.

2016-06-30361 citations

Pupillometric analysis for assessment of gene therapy in Leber Congenital Amaurosis patients.

2012-07-1923 citations

Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial.

2009-10-23635 citations

Safety and efficacy of gene transfer for Leber's congenital amaurosis.

2008-04-271586 citations